When evaluating the significance of some intervention, randomized-control trials are our gold standard. However, randomized-control trials are usually constrained by time and hindered by some bias. In ...

Rett syndrome is a rare genetic disease affecting 1 in 10,000 newborn girls, characterised by sudden regression around 1 year of age, with loss of acquired language and motor skills and leading to ...

Rett syndrome is a neurodevelopmental disorder that primarily affects girls, most of whom have mutations in the transcription regulatory gene MECP2. However, mutations in MECP2 also have been ...

Dosed First Patient in REVEAL Pivotal Trial in the Fourth Quarter of 2025. Additional enrollment continues to advance across ...

DALLAS, Jan. 22, 2024 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (TSHA), a clinical-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment ...

SAN DIEGO--(BUSINESS WIRE)--Acadia Pharmaceuticals, Inc. (Nasdaq: ACAD) today announced that interim data from the open-label real-world LOTUS™ study will be presented at the 2024 International Rett ...

Recently disclosed clinical cohort data from high (1x1015 total vg) and low dose (5.7x1014 total vg) TSHA-102 from REVEAL adolescent/adult and pediatric Phase 1/2 trials Caregiver research regarding ...

SAN DIEGO--(BUSINESS WIRE)--Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced that Health Canada has granted marketing authorization of DAYBUE™ (trofinetide) for the treatment of Rett ...

Partnership focused on utilizing Axiomer™ to develop editing oligonucleotides targeting an underlying genetic variant that causes Rett syndrome Collaboration expands ProQR’s commitment to advance the ...

First patient dosed in REVEAL pivotal trial evaluating TSHA-102 (N=15, aged 6 to Reached written alignment with FDA on inclusion of =3 months of safety data from ASPIRE trial evaluating TSHA-102 (N=3 ...